InteliSpark client, PK Biosciences Corporation has been awarded a grant from the National Institute of Neurological Disorders and Stroke (NINDS) for their project “Development of novel metformin analog for treatment of Parkinson’s Disease”. Parkinson’s disease (PD) affects more than a million Americans. Patients suffer from severe neurological deficits which become incapacitating within 10-20 years of diagnosis. Current treatments fall short in that they fail to stop the progression of the disease, rather, the existing PD treatments focus on alleviating motor symptoms by compensating for neurochemical deficits. The lack of effective neuroprotective drugs is primarily attributed to a limited understanding of the mechanisms underlying the degeneration of the nigral dopaminergic system. Furthermore, although mitochondrial dysfunction is recognized as the overriding pathophysiological hallmark of PD, no effective treatment options are available to improve mitochondrial function.
Metformin (Met) is an FDA-approved anti-diabetic drug with a remarkable safety profile. This drug was recently found to influence metabolic and cellular processes associated with aging and the development of neurodegenerative disease. However, the usage of Met as a mitochondria-targeting therapeutic is limited by its chemical properties. PK Biosciences found a way to enhance the chemical properties by increasing the mitochondrial concentration of Met from 100 to 100-fold by attaching a lipophilic cation, triphenyl phosphonium (TPP+). The newfound compound, a mitochondria-targeted metformin called MitoMet, is seen as a promising candidate for a drug development program, focusing on generating treatments for aging-related disorders and diseases attributed to mitochondrial dysfunction.
PK Biosciences has been able to study MitoMet and found two exciting properties of the compound; it is brain bioavailable, and leads to substantially higher mitochondrial biogenesis than unmodified Met in cell culture and animal model studies. Therefore, with this grant PK Biosciences will be able to further their studies of MitoMet, and determine its beneficial ability for treatment of Parkinson’s disease.